FDA pushes orphan drug research
Biogen Idec among firms urged to try existing treatments on other diseases
WASHINGTON — Roche Holding AG, Johnson & Johnson, and Biogen Idec Inc. are being urged by US regulators to see whether existing medicines may help neglected disorders, after an incentive program failed to spark research on new therapies.
The Food and Drug Administration published a list yesterday of 235 treatments that may have benefit in rare disorders and already have marketing clearance for other uses. Identifying “low-hanging fruit’’ may compel large drug makers to look beyond common ailments with guaranteed consumer demand, said Tim Cote, head of the agency’s Office of Orphan Product Development.
The list includes Roche’s hepatitis drug Pegasys, J&J’s leukemia medicine Leustatin, and Biogen Idec’s multiple sclerosis drug Avonex.
“Large pharmaceutical companies are not as engaged as we’d like to see them be,’’ Cote said in a phone interview.
About 30 million Americans have one of 7,000 rare diseases, defined by the FDA as conditions affecting fewer than 200,000 people in the United States. Medicines developed to treat these conditions are called orphan drugs, under rules that encourage their development.
Roche’s Pegasys has orphan status for chronic myelogenous leukemia. Leustatin, made by J&J, is approved for hairy cell leukemia but has had orphan drug designation since 1990 for chronic lymphochytic leukemia and acute myeloid leukemia. Also on the list is Biogen Idec’s top-selling MS drug Avonex, which has had orphan designation since 1991 as a treatment for cutaneous t-cell lymphoma.
While Biogen Idec initially pursued Avonex in “several indications’’ the Cambridge, Mass.-based drug maker now has no plans to develop it for cutaneous t-cell lymphoma, said Kate Weiss, a company spokeswoman.
“While we’re certainly always open to looking at new indications for all of our drugs, we’re not pursuing that for that indication,’’ she said yesterday.
J&J decided not to further develop Leustatin in part because there are other drugs available for leukemia, Ernie Knewitz, a spokesman, said. J&J is studying products for other types of neglected diseases, he said.
Roche is no longer studying Pegasys in cancer, said a spokeswoman for the company’s Genentech unit.
